The Medical Letter on Drugs and Therapeutics
Asfotase Alfa (Strensiq) for Hypophosphatasia (online only)
Subscribers: Log in to read full article.  Not a subscriber?  Subscribe or purchase article.

The FDA has approved asfotase alfa (Strensiq – Alexion), a recombinant form of tissue-nonspecific alkaline phosphatase, for subcutaneous treatment of perinatal-, infantile-, and juvenile-onset hypophosphatasia. Asfotase alfa is the first treatment to be approved in the US for this rare genetic metabolic disorder.

HYPOPHOSPHATASIA — Tissue-nonspecific alkaline phosphatase is essential for mineralization of bones and teeth. Hypophosphatasia is caused by mutations in the alkaline phosphatase gene. It can present at any time (in utero to adulthood) and is highly variable in severity. Perinatal- and infantile-onset hypophosphatasia can cause deformities of the chest and underdevelopment of lungs, and is often fatal. Juvenile-onset hypophosphatasia is somewhat less severe, but growth failure, skeletal deformities, and fractures can occur. Spontaneous remission seems to occur in some young adults, but ... more      

The Medical Letter is a subscriber-funded nonprofit organization that publishes critical appraisals of new prescription drugs and comparative reviews of drugs for common diseases.

Would you like to read the rest of this article?  Gain access below.

Subscriptions to The Medical Letter on Drugs and Therapeutics include:
  • Print version published and mailed biweekly (26 issues/year)
  • Unlimited online access to current and past issues (1988 - present)
  • Mobile App for iOS, Android, and Kindle Fire
  • FREE online per issue CME/CE
Purchase this article:
Title: Asfotase Alfa (Strensiq) for Hypophosphatasia (online only)
Article code: 1499e
 Electronic, downloadable article - $45
Gain access through your organization
Ask your librarian to consider an Institutional Subscription to The Medical Letter.
Recommend to your librarian